- Thinly traded nano cap Proteostasis Therapeutics (NYSE:PTI) is up 59% premarket on light volume in response to its announcement of positive results across its three cystic fibrosis (CF) pipeline programs.
- A Phase 2 study assessing 50 mg of lead candidate PTI-428 once daily for 28 days in CF patients on a background of Vertex Pharmaceuticals' (NASDAQ:VRTX) ORKAMBI (lumacaftor/ivacaftor) showed average absolute improvements in lung function (ppFEV1) of 5.2% from baseline compared to placebo (p<0.05) and average relative improvements of 9.2% (p<0.05). The treatment effect was achieved by day 14 and maintained through day 28.
- PTI-428, a CFTR amplifier, was safe and well-tolerated with no clinically meaningful interaction with lumacaftor/ivacaftor. There were two discontinuations, both mild grade thrombocytopenia, one on ORKAMBI alone and one on PTI-428. Both resolved spontaneously.
- Initial data from a 14-day study of 100 mg of PTI-801 once daily in four CF patients on background ORKAMBI showed a similar pharmacokinetic profile to healthy volunteers. There were no clinically meaningful drug-drug interactions with lumacaftor or ivacaftor. Average absolute improvements in lung function (ppFEV1) were ~4% from baseline while average relative improvements were ~7% after two weeks of dosing. Enrollment of 20 patients should be completed next quarter. PTI-801 is a CFTR corrector.
- A study of PTI-808, a CFTR potentiator, in 48 healthy volunteers showed favorable safety and pharmacokinetic profiles.
- Co-administration of PTI-428, PTI-808 and PTI-801 in 20 healthy volunteers has been completed. The safety profile was favorable and supports potential once daily dosing.
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